Summary

Eligibility
for people ages 1-17 (full criteria)
Location
at UCSF
Dates
study started
study ends around

Description

Summary

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).

Official Title

A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension

Details

This Phase 2/3 plus open-label extension study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic CAH. Part A is a Phase 2, open-label, semi-sequential cohorts portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is the open-label extension (OLE) portion of the study. Participants in Part A and B are eligible to enroll in Part C (OLE).

A total of approximately 153 participants may be enrolled in the study (planned and optional cohorts) ages 1 to < 18 years old.

The first 3 cohorts in Part A are for ages 12 to <18 years and will be semi-sequential, and Safety Review Committee (SRC) review of data and approval to proceed is required prior to enrolling each subsequent cohort. The fourth cohort in Part A is for ages 1 to 11 years old and will begin after Cohorts 1 and 2 have been completed, additional requirements are fulfilled, and following SRC review of Cohorts 1 and 2 data.

Keywords

Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia, CAH, CRN04894, Atumelnant, Pediatric, Balance-CAH

Eligibility

You can join if…

Open to people ages 1-17

Part A and B participants are eligible to be included in the study only if all of the following criteria apply:

  1. Male or female at birth, between 1 to <18 years of chronological age at the time of signing the Informed Consent Form (ICF).
  2. Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency (21-OHD) based on standard medically accepted criteria such as elevated 17-OHP level, confirmed CYP21A2 genetic testing, positive newborn screening with confirmatory second tier testing, or cosyntropin stimulation.
  3. Participants must have an elevated morning serum A4 level >ULN during Screening obtained prior to morning glucocorticoid (GC) administration.
  4. Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening.
  5. Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period.
  6. Biochemical euthyroidism as determined by the Investigator.

Part C inclusion criteria require participants to complete treatment in either Part A or Part B and in the Investigator's opinion it would benefit the participant to continue in Part C, regardless of age.

You CAN'T join if...

Part A and Part B: Individuals in Part A and Part B who meet any of the following criteria will be excluded from participation in this study:

  1. Diagnosis of any form of CAH other than classic 21-OHD.
  2. Participants treated with other GCs within 30 days of Screening.
  3. Stress dose of GC therapy within 2 weeks of start of Screening, defined as any dose above the normal maintenance dose, including but not limited to intravenous (IV) or intramuscular (IM) hydrocortisone.
  4. Use of growth hormones within 1 week of start of Screening for short acting, or within 6 weeks of start of Screening for long acting.
  5. Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening.
  6. History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ.
  7. Abnormal sleep/wake cycles (as determined by the Investigator).
  8. Female participants who are pregnant or lactating.
  9. Participants who have been dosed with an investigational drug (including atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.
  10. Individuals in Part C who do not meet the Part C Inclusion Criteria.

Locations

  • UCSF Ron Conway Gateway Medical Building accepting new patients
    San Francisco California 94158 United States
  • Cook Children's Health Care System accepting new patients
    Fort Worth Texas 76104 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Crinetics Pharmaceuticals Inc.
ID
NCT07159841
Phase
Phase 2/3 research study
Study Type
Interventional
Participants
Expecting 153 study participants
Last Updated